January 31, 2013 — Belgian biotechnology company, Cardio3 BioSciences (C3BS), a developer of regenerative and protective therapies for the treatment of cardiac diseases, announced it has received authorization from the Belgian Federal Agency for Medicines and Health Products (FAMHP) to begin its Congestive Heart failure Cardiopoietic Regenerative Therapy (CHART-1) European phase III trial for C3BS-CQR-1 in Belgium. This will be the first time a regenerative medicine targeting heart failure will be tested in a phase III trial. C3BS-CQR-1 is an autologous stem cell therapy for heart failure.

The phase III trial is a prospective, multicenter, randomized, sham-controlled, patient-and evaluator-blinded study comparing treatment with C3BS-CQR-1 to a sham treatment. The trial will recruit a minimum of 240 patients with chronic advanced symptomatic heart failure. The primary endpoint of the trial is a composite endpoint including mortality, morbidity, quality of life, six-minute walk test and left ventricular structure and function at nine months post-procedure.

Studies in additional countries will commence once national regulatory approvals have been received.

The Cardio3 BioSciences therapy, called C3BS-CQR-1, involves taking stem cells from a patient's own bone marrow and through a proprietary process called Cardiopoiesis, reprogramming those cells so that they go on to become heart cells. The cells, known as cardiopoietic cells, are then injected back into the patient's heart through a minimally invasive procedure using a catheter called C-Cathez, with the aim of repairing damaged tissue and improving heart function and patient clinical outcomes. C3BS-CQR-1 is the outcome of multiple years of research conducted at Mayo Clinic in Rochester, Minn., Cardio3 BioSciences in Mont-Saint-Guibert, Belgium, and Cardiovascular Center in Aalst, Belgium. This phase III trial builds on the successful outcome of the phase II trial conducted between 2009 and 2010 in multiple clinical sites in Belgium, Serbia and Switzerland.

"Heart failure remains a significant unmet clinical need associated with high morbidity, mortality and escalating healthcare costs. We believe C3BS-CQR-1 has a potential to become an alternative to heart transplantation, which is the only curative treatment for heart failure available today,” said Dr. Christian Homsy, CEO of Cardio3 BioSciences. “Our phase III trial is the first to begin anywhere in the world for a regenerative therapy for this indication. We look forward to confirming the promising results we have already seen in our phase II study."

"Regenerative therapies have the potential to revolutionize the treatment of heart disease and other conditions, and we are excited to be working with Cardio3 BioSciences on what is a groundbreaking study. The results seen in the earlier trials were encouraging and, if repeated in this larger study, would bring nearer a potentially disruptive treatment for the expanding epidemic of heart failure,” added Dr. Jozef Bartunek, principal investigator of the study.